Abstract | PURPOSE OF REVIEW: RECENT FINDINGS: The recent systematic review of the literature showed poor long-term outcome, with 10-year survival estimates of only 23% in 109 patients with dyskeratosis congenita who received HSCT. Multivariate analysis identified age greater than 20 years at HSCT, HSCT before 2000, and alternative donor source to be poor prognostic markers. HSCT for dyskeratosis congenita is characterized by a marked decline in long-term survival because of late deaths from pulmonary complications. However, a prospective study using danazol showed promising results in gain in telomere length and hematologic responses. SUMMARY:
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Authors | Shaimaa Elmahadi, Hideki Muramatsu, Seiji Kojima |
Journal | Current opinion in hematology
(Curr Opin Hematol)
Vol. 23
Issue 6
Pg. 501-507
(11 2016)
ISSN: 1531-7048 [Electronic] United States |
PMID | 27607446
(Publication Type: Journal Article, Review, Systematic Review)
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Topics |
- Combined Modality Therapy
- Disease Management
- Dyskeratosis Congenita
(diagnosis, etiology, mortality, therapy)
- Graft vs Host Disease
(etiology, prevention & control)
- Hematopoietic Stem Cell Transplantation
(adverse effects, methods)
- Humans
- Tissue Donors
- Transplantation Conditioning
(adverse effects, methods)
- Transplantation, Homologous
- Treatment Outcome
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